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    Summary
    EudraCT Number:2021-004156-42
    Sponsor's Protocol Code Number:ACT16970
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2022-01-20
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2021-004156-42
    A.3Full title of the trial
    A Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of SAR443820 in adult participants with
    amyotrophic lateral sclerosis, followed by an open-label extension
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2 study for SAR443820 in participants with amyotrophic lateral sclerosis (ALS)
    A.4.1Sponsor's protocol code numberACT16970
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1263-5766
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSanofi-aventis recherche & développement
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSanofi-Aventis recherche & développement
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi-aventis France
    B.5.2Functional name of contact pointDirection des Opérations Cliniques
    B.5.3 Address:
    B.5.3.1Street Address1 avenue Pierre Brossolette
    B.5.3.2Town/ cityChilly-Mazarin
    B.5.3.3Post code91385
    B.5.3.4CountryFrance
    B.5.4Telephone number0800222555
    B.5.6E-mailPublic-Registry-MA-France@sanofi.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code SAR443820
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 2252271-93-3
    D.3.9.2Current sponsor codeSAR443820
    D.3.9.3Other descriptive nameRA15804589, C19061501-F, DNL788, DN2489, DN0002489
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic lateral sclerosis
    E.1.1.1Medical condition in easily understood language
    Amyotrophic lateral sclerosis
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A
    To assess the effect of SAR443820 compared to placebo in reducing ALS progression as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
    Part B
    To assess the long-term effects of SAR443820 on function and survival
    E.2.2Secondary objectives of the trial
    Part A
    - To assess the effect of SAR443820 compared to placebo on a combined assessment of function and survival, respiratory function, muscle strength, and quality of life (QoL)
    - To assess the pharmacodynamic (PD) effect of SAR443820 compared to placebo on a key disease biomarker
    - To assess the safety and tolerability of SAR443820 compared to placebo
    - To assess the pharmacokinetics (PK) of SAR443820
    Part B
    - To assess the long-term effects of SAR443820 on disease progression, survival, respiratory function, and quality of life (QoL)
    - To assess the long-term effect of SAR443820 on a key disease biomarker
    - To assess the long-term safety and tolerability of SAR443820
    - To assess the pharmacokinetics (PK) of SAR443820
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Type of participant and disease characteristics
    • Male or female, 18-80 years of age (inclusive)
    • Diagnosis of possible, clinically probable ALS, clinically probable laboratory-supported ALS, or clinically definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria
    • Time since onset of first symptom of ALS ≤2 years.
    • Slow Vital Capacity (SVC) ≥60% of the predicted value.
    • Be able to swallow the study tablets at the screening visit.
    • Either not currently receiving riluzole or on a stable dose of riluzole for at least 4 weeks before the screening visit. Participants receiving riluzole are expected to remain on the same dose throughout the duration of the study.
    • Either not currently receiving edaravone or on the approved standard schedule of edaravone treatment. Participants receiving edaravone must have completed at least 1 cycle of treatment before the screening visit and are expected to continue edaravone treatment throughout the duration of the study.
    Weight :
    Participants with a body weight no less than 45 kg and body mass index no less than 18 kg/m2.

    Female participants with childbearing potential are eligible to participate if they are not pregnant or breastfeeding and agree to use adequate contraceptive method during study intervention period and for at least 32 days after the last dose of study drug.

    Male participants must agree to use highly effective contraceptive method during the study period and for at least 92 days following their last dose of the study drug. Male participants must not donate sperms for the duration of study and 92 days after last dose of study drug.
    E.4Principal exclusion criteria
    • A history of seizure (History of febrile seizure during childhood is allowed).
    • Having central IV lines, such as a peripherally inserted central catheter (PICC) or midline or port-a-cath lines.
    • With significant cognitive impairment, psychiatric disease, other neurodegenerative disorder (eg, Parkinson disease or AD), substance abuse, or any other condition that would make the participants unsuitable for participating in the study or could interfere with assessment or completing the study in the opinion of the Investigator.
    • History of recent serious infection (eg, pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with IV antibiotics, antivirals, or antifungals within 4 weeks of screening; or chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the Investigator’s judgment.
    • With active herpes zoster infection within 2 months prior to the screening visit.
    • A documented history of attempted suicide within 6 months prior to the screening visit, present with suicidal ideation of category 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS), or in the Investigator’s judgment are at risk for a suicide attempt.
    • History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or another medically significant illness other than ALS precluding their safe participation in this study.
    • Participants who are pregnant or are currently breastfeeding.
    • A known history of allergy to any ingredients of SAR443820.

    Prior/concomitant therapy :
    • Currently or previously treated with any strong or moderate CYP3A4 inhibitors or strong CYP3A4 inducers listed in Appendix 10 of the protocol within the specified washout period before the screening visit.
    • Received a live vaccine within 14 days before the screening visit.
    • Participants with concurrent participation in any other interventional clinical study or who have received treatment with another investigational drug
    within 4 weeks or 5 half-lives of the investigational agent before the screening visit, whichever is longer.
    • Participants who have received stem cell or gene therapy for ALS at any time in the past.
    • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3.0 × upper limit of normal (ULN)
    • Bilirubin >1.5 × ULN unless the participant has documented Gilbert syndrome (isolated bilirubin >1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin is <35%)
    • Serum albumin <3.5 g/dL
    • Estimated glomerular filtration rate <60 mL/min/1.73 m2 (Modification of Diet in Renal Disease [MDRD])

    The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
    E.5 End points
    E.5.1Primary end point(s)
    1) Change from baseline in the ALSFRS-R total score -Part A
    2) Combined assessment of the function and survival (CAFS) score -Part B
    E.5.1.1Timepoint(s) of evaluation of this end point
    1)From baseline to Week 24
    2)Week 52
    E.5.2Secondary end point(s)
    1) Combined assessment of the function and survival (CAFS) score -Part A
    2) Change from baseline in slow vital capacity (SVC) -Part A
    3) Muscle Strength – Part A
    4) Change from baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-5) -Part A
    5) Change from baseline in serum neurofilament light chain (NfL) -Part A
    6) Number of patients with treatment-emergent adverse events (TEAE) and Serious adverse event (SAE) – Part A
    7) Assessment of pharmacokinetic parameter - Plasma concentration of SAR443820 -Part A
    8) Combined assessment of the function and survival (CAFS) score – Part B
    9) Change from baseline in the ALSFRS R total score-Part B
    10) Time from baseline to the occurrence of either death, or permanent assisted ventilation (>22 hours daily for >7 consecutive days), whichever comes first – Part B
    11) Time from baseline to the occurrence of death- Part B
    12) Change from baseline in slow vital capacity (SVC)-Part B
    13) Change from baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-5)-Part B
    14) Change from baseline in serum neurofilament light chain (NfL)-Part B
    15) Number of patients with treatment emergent adverse events (TEAE) and Serious adverse event (SAE) -Part B
    16) Assessment of pharmacokinetic parameter Plasma concentration of SAR443820 -Part B
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Week 24
    2) From baseline to Week 24
    3) Over 24 Weeks
    4) From baseline to Week 24
    5) From baseline to Week 24
    6) Up to Week 24
    7) Day 1, Week 2, Week 8
    8) Week 76, Week 104
    9) From baseline to Week 52 and Week 76 and Week 104
    10) From baseline up to Week 52, Week 76 and Week 104
    11) From baseline to Week 52, Week 76 and Week 104
    12) From baseline to Week 52, Week 76 and Week 104
    13) From baseline to Week 52, Week 76 and Week 104
    14) Week 52
    15) Up to Week 106
    16) Week 28
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    Canada
    China
    France
    Germany
    Italy
    Japan
    Netherlands
    Spain
    Sweden
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 85
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 176
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state24
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 261
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-03-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-01-18
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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